Arizona Center for Innovation (AzCI) presents: Working with Your Demographic Market (in orphan drug development) This presentation is part of a series developed for a workshop on "How to Navigate the Biotech Regulatory Process" The Arizona Center for Innovation is an incubator and innovation center and provides resources in support of startups getting to the next level and become successful enterprises.

2. ARIZONA
CENTER

3. Innovation Center
The Arizona Center for Innovation (AzCI) assists
technology companies turning their innovative ideas
into successful businesses through:
• Focused programs
• World-class expertise
• High-quality facilities
Access:
• Other technology
entrepreneurs
• Collaborative,
creative
environment
• Advantage of
hands-on support
• Successful
business leaders
Arizona Center for Innovation

4. AzCI
Startup and emerging technology companies
• Community and UA spin-outs
Technology
• Aligned with UA Tech Parks and UA
International
• Softlandings
Who

5. AzCI
Technology Areas:
• Security and Defense
• Mining
• Agriculture and Water
• Biotechnology
• Intelligent Transportation and Vehicles
• Renewable Energy
Informatics/Big Data/Advanced Manufacturing/
Imaging/Optics/Photonics
Focus

6. Working withYour
Demographic Market
Arizona Center for Innovation

7.  Locate the patients
 Identify key physicians
 Patient groups
 Key meetings

9.  Industry
 Patients
 Academia/physicians
 Government

10. Finding Patients

11.  Internet
 Mapping projects
 Patient Organizations
 Key Opinion Leaders
 Physician meetings
 Rare disease resources
 Clinical Research Organizations

12.  Background information on the disease
 WebMd, Medline Plus, CDC, Healthfinder, NIH
 News sources- press releases, features
 Research in the area- institutions
 Publications and presentations –researcher
presentations
 Current therapies
 EMAUD – European Markets Access
University Diploma

13.  Facebook
 Twitter
 Listservs
 Linked In
 Check Orphan
 Yahoo

14.  Effective tool for smaller patient groups that
are treated by a group of specialists
 Develop demographic survey
 Number of patients
 Physical status
 Eligibility for a clinical trial
 Interest of physician in trial participation
 Creates a database of where patients are and
physicians interested in trial participation

15.  Internet
 Publications
 Affiliations
 Memberships
 Specialty organizations
 Physician advisory board for patient group
 Department chairman

16.  Don’t hesitate to contact- email first
 Generally connected to patient groups
 May be board member of patient group
 Know other key treaters
 Researchers will have colleagues around the
world
 Generally attend key meetings in their
specialty area

17.  Local, national, international
 Frequency of meeting
 Conference agenda
 Conference symposiums
 Special interest groups
 Conference proceedings

18.  Orphanet –European focused
 Prevalence of disease
 Location of clinical trials
 Patient organizations
 Research centers
 NORD- US based
 Many support group members
 Good networking for industry, FDA, NIH, and
patients

19.  Eurordis –European Patient Organizations
 Disease and drug information
 Educational programs
 Represent national and pan European groups
 GeneticAlliance- US based
 Focus on genetic diseases
 Large biomarker/ family history sites
 Annual patient meeting

20.  FDA Office of Orphan Products
 Orphan drug designation
 Research grants
 Protocol assistance
 NIH Office of Rare Diseases
 Research funding
 Educational programs

21.  Understand their evolution and mission
 Advocacy
 Education
 Support
 Financial support
 Level of organization
 Level of sophistication

22.  Traditional Pharma
 Sales force promotes products
▪ Samples
▪ Physician office visits
▪ Educational programs
 Let me tell you why you
should buy a new red
wagon
 Customer may see only a
limited use for the wagon

23.  Orphan Pharma advocates
 Small or non-existent sales force
 Medical Science Liaisons
 Focus on direct patient and physician
relationships
 My red wagon is big
enough for two
 We can explore the world in
in our red wagon
 My Dad likes pulling us

24.  Rule #1 Include patients from the beginning
 Rule #2 Repeat rule number one

25. Rangnekar, Dwijen
Patient Groups and the Innovation Process
Drug Proof of Clinical Trials and Regulatory Drug
Discovery Principle Development Approval Consumption
Participating in and
designing clinical trials
Funding and
stimulating
medical
research
Drug uptake
and market
Regulatory approval
Patient Groups

26.  Invited speakers
 PatientAdvisory Boards
 Local, regional, national, global
 Age
 Severity of illness
 Parents, siblings
 Attendance at patient meetings

27.  Identify KOLs
 Identify patients through advertisements,
social media
 Identify potential clinical sites
 Have large patient databases

28.  Patients know other patients often all over
the world
 Patients communicate with each other
 Patients and doctors communicate with each
other
 Patients are quick to tell other patients about
new treatments

29.  Over 1200 disease specific groups listed in
the NORD directory
 Most created since the 1980’s
 AIDS support groups’ success spurred
development of many other groups

30.  Different relationship than with big
pharma
 Small number of patients and physicians
 Personal relationship with patients and
physicians
 More dependent on pharma for financial
support
 May only have one company to provide
support

31.  Promote drugs and disseminate
information more freely than through
the company
 Spread awareness of new drugs at a pre-
launch stage without critical review
 Provide more credible endorsement of
products than the company
 Can act as advocates to government
agencies

32.  Funding and business “know how”
 Access to key people in pharma
 Access to newest research information
 Free publicity
 Production of information materials
 Lobbying for resources to help members

33.  Support groups have adversarial role to
pharma
 Pharma initiates support groups to create
drug market
 Pharma supports creation of databases to
further its interests

34. AcademiaPatients
Government
Industry

35. Stool is only as strong as its
weakest leg

36. https://techparks.arizona.edu/azci
©Arizona Center for Innovation 2015